Phase 1 trial

The first healthy volunteer has been enrolled in a Phase 1 study testing ABX1100 — a novel substrate reduction therapy from Aro Biotherapeutics — as a treatment for Pompe disease. This will be the first-in-human trial for the experimental therapy, meaning it’s the first time ABX1100 has been…

Single and multiple ascending doses of MZE001, Maze Therapeutics‘ experimental oral substrate reduction treatment for Pompe disease, were well tolerated and reduced glycogen levels in healthy volunteers in a Phase 1 trial, data show. These findings support a Phase 2 trial of MZE001 in people with Pompe disease,…

MZE001, an experimental oral substrate reduction treatment for Pompe disease, was well-tolerated in both single and multiple doses in a study conducted in healthy volunteers, according to Maze Therapeutics, the therapy’s developer. Based on the trial’s findings, Maze is planning to launch a new study in 2023 to…

Duke Health researchers are planning to launch a Phase 1 trial to test the safety of a new investigational gene therapy they developed to treat patients with Pompe disease. They are currently screening (NCT03285126) adults with late-onset Pompe disease to select eligible participants for the upcoming trial.